Corporate Banner
Satellite Banner
Stem Cells, Cellular Therapy & Biobanking
>
Scientific Community
 
Become a Member | Sign in
Home>News>This Article
  News
Return

Promethera Successfully Enrolls Twenty Patients in its Multicentric Phase I/II Trial

Published: Tuesday, November 12, 2013
Last Updated: Tuesday, November 12, 2013
Bookmark and Share
Promethera Biosciences has conducted the trial in Belgium, France, United Kingdom, Italy and Israel, treating twenty patients affected by very rare liver-based metabolic diseases.

Promethera Biosciences has announced the completion of patient enrolment for its first clinical trial.

Promethera Biosciences is conducting a first-in-man trial with HepaStem in paediatric patients suffering from orphan diseases.

This trial is a prospective, open label, multicenter phase I/II clinical study involving Urea Cycle Disorders (UCD) and Crigler-Najjar (CN) syndrome paediatric patients. The trial is a dose escalation study designed to evaluate the safety and the preliminary efficacy of Promethera HepaStem.

Most young patients affected by these very rare diseases have limited therapeutic options and may die at an early age. CN syndrome has an incidence of around one in a million births. It becomes apparent during the neonatal period by early intense jaundice due to unconjugated bilirubin.

The UCD are a group of eight inborn errors of metabolism that affect the transfer of nitrogen into urea. Although each specific disorder results in the accumulation of different precursors, hyperammonemia and hyperglutaminemia are common biochemical hallmarks of these disorders.

UCD are also considered as rare diseases with an incidence between 1 in 8,000 and 1 in 44,000.

Promethera Biosciences has successfully treated 20 patients with HepaStem in collaboration with 11 clinical centers. The end of the trial is scheduled for October 2014 after a patient follow-up period of 12 months.

Promethera HepaStem is an innovative cell therapy product based on the use of allogeneic progenitor cells derived from livers obtained from adult (non-embryonic) organ donors. These cells are capable of in-vitro expansion and in-vivo differentiation into hepatocyte-like cells. The cells can be used to treat a wide variety of liver diseases from rare inborn metabolic diseases such as UCD and CN (classified as ‘orphan diseases’ and mainly affecting children), to acquired deficiencies affecting adults such as fulminant hepatitis or liver fibrosis.

Given the scarcity of patients, Promethera Biosciences has conducted the trial in five countries: Belgium, France, United Kingdom, Italy and Israel. In total, 11 clinical centers actively participate in the study: Cliniques Universitaires Saint-Luc, Belgium, Universitair Ziekenhuis Antwerp, Belgium, Hopital Jeanne de Flandre in Lille, France, Centre Hospitalier Universitaire Bicetre in Paris, France, Centre Hospitalier Universitaire de Toulouse, France, Birmingham Children’s Hospital, UK, Great Ormond Street Hospital, UK, IRCCS Ospedale Pediatrico del Bambino Jesu, Italy, Meyer Children’s Hospital at Rambam Health Care Campus, Israel, Hadassah Ein-Kerem Medical Center, Israel, Schneider Children’s Medical Center of Israel. The study started in March 2012 with the first patient treated in Belgium. As of today, 20 patients aged from six weeks to 16 years have been treated with Promethera HepaStem.

Dr. Beatrice De Vos, chief medical officer of Promethera Biosciences said: “The successful recruitment of 20 patients suffering from very rare diseases in only 18 months was achieved thanks to three important factors: an extensive network of collaborating centers of the founder hospital, strong communication with the treating physicians of the patients and dedicated teams at Promethera Biosciences. Each new country and each new clinical center brought new challenges that our teams have addressed inventively and professionally.”

Professor Etienne Sokal, chief scientific officer of Promethera Biosciences, paediatric hepatologist at Cliniques Universitaires Saint Luc, Belgium and director of UCL’s Liver Cell Therapy Program, Belgium, said: “With patients treated at their own center, separate from Promethera, we have demonstrated the feasibility of proposing HepaStem treatment to any patient around the world, giving them the opportunity to receive innovative treatment locally.”

Eric Halioua, chief executive officer of Promethera Biosciences said: “We are very pleased to have achieved this new milestone in the development of our main product HepaStem. During the trial another innovation took place with the use of a mobile formulation unit operating near the medical site. This mobile production unit was used to both carry the material and to formulate the product. The GMP approval process by the Belgian authorities considered the unit as an extension of the Promethera Biosciences GMP certified manufacturing unit. A sterile and closed formulation system for preparing the final product was installed in this unit, operating so that the cells could be delivered in real time at the bedside of the patient. A patent for the mobile unit has been filed and is being examined before issuance.”


Further Information
Access to this exclusive content is for Technology Networks Premium members only.

Join Technology Networks Premium for free access to:

  • Exclusive articles
  • Presentations from international conferences
  • Over 2,400+ scientific posters on ePosters
  • More than 3,700+ scientific videos on LabTube
  • 35 community eNewsletters


Sign In



Forgotten your details? Click Here
If you are not a member you can join here

*Please note: By logging into TechnologyNetworks.com you agree to accept the use of cookies. To find out more about the cookies we use and how to delete them, see our privacy policy.

Related Content

Promethera Receives Approval to Enroll Patients in the Imminent HEP002 Phase IIb/III Clinical Trial
HEP002 trial represents the world’s first Phase IIb/III trial with a stem cell-based medicinal product for the treatment of urea cycle disorders patients.
Thursday, October 16, 2014
Promethera Biosciences Announces CFO
Frank Hazevoets joined the Company on May 1, 2014 as chief financial officer.
Tuesday, May 13, 2014
Promethera Biosciences Treats First Patients with Promethera® HepaStem
Phase I/II study designed to evaluate the ability of liver progenitor cells to restore a missing enzyme function in liver-based metabolic diseases.
Tuesday, May 15, 2012
Scientific News
New Weapon in the Fight Against Blood Cancer
This strategy, which uses patients’ own immune cells, genetically engineered to target tumors, has shown significant success against multiple myeloma, a cancer of the plasma cells that is largely incurable.
Scientists Create CRISPR/Cas9 Knock-In Mutations in Human T Cells
In a project spearheaded by investigators at UC San Francisco, scientists have devised a new strategy to precisely modify human T cells using the genome-editing system known as CRISPR/Cas9.
Zebrafish Reveal Drugs that may Improve Bone Marrow Transplant
Compounds boost stem cell engraftment; could allow more matches for patients with cancer and blood diseases.
New Material Forges the Way for 'Stem Cell Factories'
Researchers have discovered the first fully synthetic substrate with potential to grow billions of stem cells. The researchcould forge the way for the creation of 'stem cell factories' - the mass production of human embryonic (pluripotent) stem cells.
Liver Regrown from Stem Cells
Scientists have repaired a damaged liver in a mouse by transplanting stem cells grown in the laboratory.
Immunotherapy Shows Promise for Myeloma
A strategy, which uses patients’ own immune cells, genetically engineered to target tumors, has shown significant success against multiple myeloma, a cancer of the plasma cells that is largely incurable.
'Google Maps' for the Body
Scientists have revealed research that uses previously top-secret technology to zoom through the human body down to the level of a single cell that could be a game-changer for medicine.
Adaptimmune's Novel Cancer Therapeutics Show Positive Clinical Trial Results
The company has announced that positive data from its Phase I/II study of its affinity enhanced T-cell receptor (TCR) therapeutic targeting the NY-ESO-1 cancer antigen in patients with multiple myeloma has been published.
Stem Cells Rescue Patients from Mitochondrial Disease
A study led by OHSU researchers has revealed a critical first step in developing a new gene and stem cell regenerative technique for treating patients with mitochondrial disease.
Eco-Friendly Nanobullet to Battle Bacteria
Researchers have developed a method to combat bacteria by engineering nanoscale particles that add the antimicrobial potency of silver to a core of lignin, a ubiquitous substance found in all plant cells.
Skyscraper Banner

Skyscraper Banner
Go to LabTube
Go to eposters
 
Access to the latest scientific news
Exclusive articles
Upload and share your posters on ePosters
Latest presentations and webinars
View a library of 1,800+ scientific and medical posters
2,400+ scientific and medical posters
A library of 2,500+ scientific videos on LabTube
3,700+ scientific videos
Close
Premium CrownJOIN TECHNOLOGY NETWORKS PREMIUM FREE!